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Wiener klinische Wochenschrift

Erschienen in:

12.04.2022 | original article

Long-term eliglustat treatment of Gaucher patients over up to 10 years in Vienna

verfasst von: Thomas M. Stulnig

Erschienen in: Wiener klinische Wochenschrift | Ausgabe 11-12/2022

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Summary

Gaucher disease has been the first lysosomal storage disorder for which an enzyme replacement therapy has been approved in the 1990s and was the first to receive approval for a first-line substrate reduction therapy in 2015. Eliglustat treatment has been started in Austria in patients recruited to a clinical trial, followed by its long-term extension and prescription treatment overall covering up to 10 years. In this case series the experience of treating Gaucher patients with eliglustat in Vienna is summarized. Patients were either switched from enzyme replacement therapy or were therapy naïve. Significant improvements were shown in hematological (thrombocytes, hemoglobin) and visceral (spleen volume) manifestations as well as in biomarkers (chitotriosidase, glucosylsphingosine [lyso-GL1], angiotensin converting enzyme) in a routine setting in a therapy-naïve patient. Stability was found in switch patients with slight improvement in bone density. Eliglustat was generally very well tolerated. Patient selection and regular monitoring is required to ensure effective and safe use.

Vorheriger Artikel Association of HLA-DRB1 alleles with rheumatoid arthritis in Split-Dalmatia County in southern Croatia

Nächster Artikel Successful treatment of acquired von Willebrand syndrome associated with monoclonal gammopathy

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Titel: Long-term eliglustat treatment of Gaucher patients over up to 10 years in Vienna
verfasst von: Thomas M. Stulnig
Publikationsdatum: 12.04.2022
Verlag: Springer Vienna
Erschienen in: Wiener klinische Wochenschrift / Ausgabe 11-12/2022
Print ISSN: 0043-5325
Elektronische ISSN: 1613-7671
DOI: https://doi.org/10.1007/s00508-022-02021-2

Springer Medizin Österreich

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